BrainStorm Cell Therapeutics Announces Support for FDA Guidelines on ALS Drug Development
September 27 2019 - 4:00AM
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the
development of innovative autologous cellular therapies for highly
debilitating neurodegenerative diseases supports the US FDA’s
publication on September 24, 2019 of “Amyotrophic Lateral
Sclerosis: Developing Drugs for Treatment Guidance for Industry.”
This document outlines ALS drug development guidelines and
potential opportunities to advance novel ALS therapies through
innovative clinical trial design and validated clinical outcome
measures.
In addition, Brainstorm’s President & CEO
Chaim Lebovits, COO & CMO Ralph Kern MD MHSc, and Mary Kay
Turner, VP Government Affairs and Patient Advocacy, convened a
roundtable discussion with ALS advocacy group leaders in
Washington, DC, on September 25, 2019. Members of The US Congress
and their Staff, including the Staff of the Co-Chairs of the ALS
Caucus, attended to listen to the voice of the ALS Community.
Members of the ALS Community included: ALS Association, ALS
One, Compassionate Care ALS, NoMoreExcuses, ALS Therapy Development
Institute, Paralyzed Veterans of America, IamALS, and Project ALS.
The Roundtable was convened for the ALS Community to learn more
about Brainstorm’s NurOwn® phase 3 clinical development program in
ALS and to ensure the patient voice was an ongoing, integral part
of the discussion.
Chaim Lebovits, President and CEO of BrainStorm
stated, “The updated guidelines recognize the serious and
life-threatening nature of ALS and enormous unmet need. We are
encouraged that the FDA has updated the ALS guidance document and
we commend the advocacy community for keeping the patient voice at
the center of this dialogue.” Mr. Lebovits added, “The guidance
document proposes regulatory flexibility, the importance of
establishing clinical benefit based on functional endpoints, and
practicable study size and duration. We believe that these new
guidelines are essential in order to develop critically needed
drugs for those suffering from ALS.”
Mary Kay Turner, Vice President of Government
Affairs and Patient Advocacy of BrainStorm stated, “Now is such a
hopeful time for the ALS Community - the Community is active,
informed and engaged in powerful ways. The roundtable discussion
was organized as a forum where representatives of the ALS Advocacy
Community, Members of the US Congress and their Staff, and the
BrainStorm team exchanged many meaningful ideas. Over the course of
several hours, challenges and hopes were expressed, and the
incredible ALS community shared their visions for a future where
ALS is no more. Having Members of Congress and their Staff join the
meeting underscores the ALS patient voice is being heard in a
powerful and very meaningful way.”
About NurOwn® NurOwn®
(autologous MSC-NTF) cells represent a promising investigational
therapeutic approach to targeting disease pathways important in
neurodegenerative disorders. MSC-NTF cells are produced from
autologous, bone marrow-derived mesenchymal stem cells (MSCs) that
have been expanded and differentiated ex vivo. MSCs are converted
into MSC-NTF cells by growing them under patented conditions that
induce the cells to secrete high levels of neurotrophic factors.
Autologous MSC-NTF cells can effectively deliver multiple NTFs and
immunomodulatory cytokines directly to the site of damage to elicit
a desired biological effect and ultimately slow or stabilize
disease progression. BrainStorm is currently conducting a Phase 3
pivotal trial of autologous MSC-NTF cells for the treatment of
amyotrophic lateral sclerosis (ALS). BrainStorm also recently
received U.S. FDA acceptance to initiate a Phase 2 open-label
multicenter trial in progressive MS and enrollment began in March
2019.
About BrainStorm Cell Therapeutics
Inc.BrainStorm Cell Therapeutics Inc. is a leading
developer of innovative autologous adult stem cell therapeutics for
debilitating neurodegenerative diseases. The Company holds the
rights to clinical development and commercialization of the NurOwn®
technology platform used to produce autologous MSC-NTF cells
through an exclusive, worldwide licensing agreement. Autologous
MSC-NTF cells have received Orphan Drug status designation from the
U.S. Food and Drug Administration (U.S. FDA) and the European
Medicines Agency (EMA) in ALS. BrainStorm is currently enrolling a
Phase 3 pivotal trial in ALS (NCT03280056), investigating
repeat-administration of autologous MSC-NTF cells at six sites in
the U.S., supported by a grant from the California Institute for
Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is
intended to support a filing for U.S. FDA approval of autologous
MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA
clearance to initiate a Phase 2 open-label multicenter trial in
progressive Multiple Sclerosis. The Phase 2 study of autologous
MSC-NTF cells in patients with progressive MS (NCT03799718) started
enrollment in March 2019. For more information, visit the company's
website at www.brainstorm-cell.com
Safe-Harbor
StatementsStatements in this announcement other than
historical data and information constitute "forward-looking
statements" and involve risks and uncertainties that could cause
BrainStorm Cell Therapeutics Inc.'s actual results to differ
materially from those stated or implied by such forward-looking
statements. Terms and phrases such as "may", "should", "would",
"could", "will", "expect", "likely", "believe", "plan", "estimate",
"predict", "potential", and similar terms and phrases are intended
to identify these forward-looking statements. The potential risks
and uncertainties include, without limitation, risks associated
with BrainStorm's limited operating history, history of losses;
minimal working capital, dependence on its license to Ramot's
technology; ability to adequately protect the technology;
dependence on key executives and on its scientific consultants;
ability to obtain required regulatory approvals; and other factors
detailed in BrainStorm's annual report on Form 10-K and quarterly
reports on Form 10-Q available at http://www.sec.gov. These factors
should be considered carefully, and readers should not place undue
reliance on BrainStorm's forward-looking statements. The
forward-looking statements contained in this press release are
based on the beliefs, expectations and opinions of management as of
the date of this press release. We do not assume any obligation to
update forward-looking statements to reflect actual results or
assumptions if circumstances or management's beliefs, expectations
or opinions should change, unless otherwise required by law.
Although we believe that the expectations reflected in the
forward-looking statements are reasonable, we cannot guarantee
future results, levels of activity, performance or
achievements.
CONTACTS
Corporate: Uri Yablonka Chief
Business Officer BrainStorm Cell Therapeutics Inc. Phone:
646-666-3188 uri@brainstorm-cell.com
Media:Sean LeousWestwicke/ICR
PR Phone: +1.646.677.1839sean.leous@icrinc.com
Brainstorm Cell Therapeu... (NASDAQ:BCLI)
Historical Stock Chart
From Aug 2024 to Sep 2024
Brainstorm Cell Therapeu... (NASDAQ:BCLI)
Historical Stock Chart
From Sep 2023 to Sep 2024